Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO) (CALLISTO)

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Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

Primary objective: to establish the pharmacokinetic profile of omigapil at a range of doses in paediatric and adolescent patients with CMD. [ Time Frame: Week 4 and Week 8 ]

Secondary Outcome Measures
:

Secondary objective: to evaluate the safety and tolerability of omigapil at a range of doses in paediatric and adolescent patients. [ Time Frame: Baseline, Week 4, 8 and 12 ]

Other Outcome Measures:

Tertiary objective: to establish the feasibility of conducting disease-relevant clinical assessments in paediatric and adolescent patients with CMD to aid in the design of future studies [ Time Frame: Baseline, Week 12 and 16 ]

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Ages Eligible for Study:

5 Years to 16 Years (Child)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old or more and less than 17 years old) at time of screening with a clinical picture (see below) consistent with Ullrich CMD or MDC1A (LAMA2/merosin deficient CMD)

• Evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy with matching clinical phenotype and no suspicion of alpha dystroglycanopathy (aDG-RD) (clinically or by staining on muscle biopsy)

Exclusion Criteria:

Use of any investigational drug other than the study medication within 12 weeks of study start.

Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)

Patients with respiratory parameters (eg: low pulmonary function test value i.e. <30% or need for brief course of daytime non-invasive ventilation) currently affected by short term medications, or acute illness/ conditions (conduct baseline assessments when the patient has recovered and no longer taking acute medication)

Any need for surgery (scoliosis, gastrostomy, other) in the preceding 24 weeks or foreseen during the course of the study.

Patient has an intercurrent significant medical condition or situation which in the opinion of the Investigator or the study Medical Monitor may put the patient at significant risk, confound the study results or interfere significantly with the patient's participation in the study

In patients below the 3rd percentile, any further drop in body weight percentile in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)

Weight less than 17kg at Baseline

Morbidly obese or grossly overweight (≥86 percentile BMI in children)

History of epilepsy or on antiepileptic medication at Screening/Baseline